The Therapeutic Potential of MicroRNAs in Cancer: Illusion or Opportunity?

Orazio Fortunato1, Marilena V Iorio2

  • 1Tumor Genomics Unit, Department of Research, Fondazione IRCCS Istituto Nazionale dei Tumori, via Venezian 1, 20133 Milan, Italy.

Insights

MicroRNAs show promise for cancer therapy by modulating gene expression. However, challenges like stability, delivery, and off-target effects must be addressed for safe and effective clinical application.

Area of Science:

  • Oncology
  • Molecular Biology
  • Biotechnology

Background:

  • MicroRNAs (miRNAs) are increasingly recognized for their role in human neoplasia.
  • The potential of modulating miRNA expression for cancer treatment is a significant area of research.

Purpose of the Study:

  • To review the challenges associated with miRNA-based cancer therapy.
  • To explore current delivery strategies for miRNA therapeutics.
  • To summarize ongoing clinical trials in miRNA-based cancer therapy.

Main Methods:

  • Literature review of miRNA function in neoplasia.
  • Analysis of miRNA stability, tissue penetration, and off-target effects.
  • Survey of existing miRNA delivery systems and clinical trial data.

Main Results:

  • miRNA-based therapies offer potential for re-establishing cellular equilibrium and enhancing treatment response.
  • Key challenges include miRNA stability in circulation, effective tissue penetration, and minimizing off-target effects.
  • Various delivery strategies are being investigated to overcome these hurdles.

Conclusions:

  • miRNA-based therapies hold promise for cancer treatment but require careful consideration of safety and efficacy.
  • Addressing delivery and stability challenges is crucial for clinical translation.
  • Ongoing clinical trials are evaluating the therapeutic potential of miRNAs in oncology.

Related Concept Videos

MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
3.4K
MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After...
23.3K
mTOR Signaling and Cancer Progression03:03

mTOR Signaling and Cancer Progression

The mammalian target of rapamycin or mTOR protein was discovered in 1994 due to its direct interaction with rapamycin. The protein gets its name from a yeast homolog called TOR. The mTOR protein complex in mammalian cells plays a major role in balancing anabolic processes such as the synthesis of proteins, lipids, and nucleotides and catabolic processes, such as autophagy in response to environmental cues, such as availability of nutrients and growth factors.
The mTOR pathway or the...
4.2K
lncRNA - Long Non-coding RNAs02:39

lncRNA - Long Non-coding RNAs

In humans, more than 80% of the genome gets transcribed. However, only around 2% of the genome codes for proteins. The remaining part produces non-coding RNAs which includes ribosomal RNAs, transfer RNAs, telomerase RNAs, and regulatory RNAs, among other types. A large number of regulatory non-coding RNAs have been classified into two groups depending upon their length – small non-coding RNAs, such as microRNA, which are less than 200 nucleotides in length, and long non-coding RNA...
9.5K
Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
6.9K
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
17.7K